JOP20190104A1 - مركبات وطرق لتقليل التعبير عن atxn3 - Google Patents

مركبات وطرق لتقليل التعبير عن atxn3

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Publication number
JOP20190104A1
JOP20190104A1 JOP/2019/0104A JOP20190104A JOP20190104A1 JO P20190104 A1 JOP20190104 A1 JO P20190104A1 JO P20190104 A JOP20190104 A JO P20190104A JO P20190104 A1 JOP20190104 A1 JO P20190104A1
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JO
Jordan
Prior art keywords
compounds
methods
reducing
animal
cell
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Application number
JOP/2019/0104A
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English (en)
Inventor
Holly Kordasiewicz
Susan M Freier
Original Assignee
Ionis Pharmaceuticals Inc
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Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of JOP20190104A1 publication Critical patent/JOP20190104A1/ar

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181Peptide nucleic acid, PNA
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/33415-Methylcytosine
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy

Abstract

يتعلق الاختراع الحالي بمركبات، وطرق، وتركيبات صيدلية لتقليل كمية أو نشاط ATXN3 mRNA في خلية أو حيوان، وفي حالات معينة تقليل كمية بروتين Ataxin-3 في خلية أو حيوان. تُعد مثل تلك المركبات والطرق والتركيبات الصيدلية مفيدة في الوقاية من أو تخفيف عرض أو علامة مميزة واحدة على الأقل لمرض تنكسي عصبي. تتضمن مثل هذه الأعراض والعلامات المميزة الترنح، الاعتلال العصبي، وتكوين التجمعات. وتشتمل مثل هذه الأمراض التنكسية العصبية على SCA3.
JOP/2019/0104A 2016-11-10 2017-06-16 مركبات وطرق لتقليل التعبير عن atxn3 JOP20190104A1 (ar)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662420294P 2016-11-10 2016-11-10

Publications (1)

Publication Number Publication Date
JOP20190104A1 true JOP20190104A1 (ar) 2019-05-07

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Family Applications (1)

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JOP/2019/0104A JOP20190104A1 (ar) 2016-11-10 2017-06-16 مركبات وطرق لتقليل التعبير عن atxn3

Country Status (21)

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US (2) US11583548B2 (ar)
EP (1) EP3538656A4 (ar)
JP (1) JP2019534009A (ar)
KR (1) KR20190076025A (ar)
CN (1) CN109923210A (ar)
AR (1) AR110878A1 (ar)
AU (1) AU2017357760A1 (ar)
BR (1) BR112019007383A2 (ar)
CA (1) CA3041347A1 (ar)
CL (1) CL2019001166A1 (ar)
CO (1) CO2019003729A2 (ar)
CR (1) CR20190277A (ar)
EA (1) EA201990862A1 (ar)
IL (1) IL266169A (ar)
JO (1) JOP20190104A1 (ar)
MA (1) MA46791A (ar)
MX (1) MX2019005506A (ar)
PE (1) PE20191047A1 (ar)
SG (1) SG11201903167QA (ar)
TW (1) TW201819397A (ar)
WO (1) WO2018089805A1 (ar)

Families Citing this family (13)

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US10533175B2 (en) 2015-09-25 2020-01-14 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating Ataxin 3 expression
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
AU2019266307A1 (en) 2018-05-09 2020-11-19 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ATXN3 expression
CN112400019B (zh) 2018-06-27 2024-05-07 Ionis制药公司 用于减少lrrk2表达的化合物和方法
CN113383077A (zh) * 2018-11-19 2021-09-10 优尼科Ip有限公司 RNAi诱导的ataxin-3减少以用于治疗脊髓小脑共济失调3型
KR20210093970A (ko) 2018-11-21 2021-07-28 아이오니스 파마수티컬즈, 인코포레이티드 프리온 발현을 감소시키기 위한 화합물 및 방법
WO2020245233A1 (en) 2019-06-06 2020-12-10 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
CN110527741A (zh) * 2019-09-11 2019-12-03 东北农业大学 一种与美洲南瓜抗白粉病生理小种2f基因紧密连锁的分子标记、引物及应用
WO2021053018A1 (en) * 2019-09-16 2021-03-25 Uniqure Ip B.V. Targeting misspliced transcripts in genetic disorders
JPWO2021230286A1 (ar) * 2020-05-12 2021-11-18
AR124229A1 (es) 2020-12-03 2023-03-01 Hoffmann La Roche Oligonucleótidos antisentido que actúan sobre atxn3
WO2022117747A2 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
AR127158A1 (es) 2021-09-29 2023-12-27 Hoffmann La Roche Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina

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